A review article dated 2 May 2022 in the American Cancer Society Journal ‘Cancer’ titled ‘Defining disease modification in myelofibrosis in the era of targeted therapy’ has outlined a promising way forward for myelofibrosis (MF) targeted therapies.

The authors are several renowned MPN specialists from around the world and their article refers to ‘exciting developments’. Their conclusions are extremely encouraging for all MPN patients.

Currently, the only cure for MF is an allogeneic stem cell transplant and treatment criteria for MF have mainly focussed on relieving symptoms rather than survival benefit.  However in this review article, the authors outline several promising clinical trials able to modify the disease course in MF.  While these trials have had relatively small patient numbers, the authors’ view is that the treatment landscape for MF is ‘set to evolve rapidly as understanding of the molecular pathogenesis of MF sheds light on novel therapeutic targets and the possibility of selectively depleting the malignant HSC compartment.’

The article concludes:
‘In summary, the possibility of disease modification has the potential to revolutionize clinical practice and treatment decision-making for patients with MF. As novel end points begin to emerge, it will be important to re-evaluate clinical trial designs, and potentially redefine disease modification, adding new end points to survival outcomes, to ensure the true potential for disease modification and MF therapy is realized. Standardized definitions and assessments are needed across clinical trials, along with the inclusion of patients with newly diagnosed disease, where the greater potential for disease modification may lie.’

The full article is unlocked and able to be read HERE.